Gene Therapy GenSight GS010 / GS011

GS010 is a form of Gene Therapy.

The actual mitochondrial gene affected by the LHON 11778 mutation is called the ND4 gene.

The unaffected or “wild” form of the ND4 Gene is combined with targeting information into and packaged into the shell of an  AAV2 virus

Once the virus is injected into the eye, it delivers the DNA into the cells.  The cells use the gene DNA to make RNA.  The RNA is used by protein-manufacturing sites on the surface of mitochondria to make unaffected copies of the ND4 protein subunit inside the mitochondria. (26029714)

This use of RNA to target the protein-making sites of mitochondria is a key feature of Gensight’s process

The unaffected version of the ND4 protein subunit combines with other proteins to form Complex I of the Respiratory Chain.


This treatment will help those with LHON mutations in the ND4 Gene, such as the G11778A mutaation, but will not be suitable for treating people affected by the 3460 mutation, which is in the ND1 Gene, or the 14484 mutation, which is in the ND6 Gene.

GenSight have a therapy containing the ND1 Gene (the 3460 LHON mutation) in their development pipeline.

GS010 development has been documented in a mouse model of LHON (22669418) and in primates (

A safety and tolerability trial in humans began in 2014.

Gensight Biologics received FDA aproval to move into Phase III Clinical Trials September 10 2015.

Gensight are about to start clinical trials in France, Germany, Italy, the UK and USA.

The RESCUE trial will recruit patients who have been affected for less than six months.

The REVERSE trial will recruit patients who have been affected between six and twelv3 months.

Clinical Trial NCT02064569

This therapy can only help cells which are still working well enough to manufacture the ND4 protein and incorporate it into working mitochondria.  That means it is aimed at protecting the remaining eyesight of someone who is in the early stages of losing eyesight due to LHON.(26029714)

We do not know if the therapy would allow any regeneration of lost or dormant optic nerve fibres, and so we do not know if any eyesight would be actually recovered as a result of this therapy.

Further information can be found at:

GenSight GS010 Page

Gensight development pipeline

This page was last updated September 17 2015





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